New technology enables the insertion of a large segment of DNA into a genome, potentially expanding gene therapy treatment from cancellation of disease-causing mutations to replacement of an entire gene, scientists say.

Reporting in Nature, the researchers describe building upon a technique called prime editing by inserting DNA that attaches to the genome through a series of overlapping flaps. This method, which they call a prime assembly approach, avoids a bottleneck in the gene therapy field—a double-strand break to the donor DNA that can cause toxicity and kill cells.

“Using this method, we are doing genome assembly rather than making a small edit in a gene,” said Bin Liu, a co-lead author of the study and assistant professor of biological chemistry and pharmacology at The Ohio State University College of Medicine. “If we think of the genome as a book, we can remove one paragraph and replace it with a new one—or even rewrite a chapter.”

Scientists have uncovered an unexpected way cells can generate cancer-driving proteins—by cutting RNA into shorter, functional fragments rather than following the standard blueprint. This process, newly termed as “RNA dicing,” enables the production of a truncated form of the JAK1 protein that remains highly active and can promote tumor growth, particularly when normal gene function is disrupted.

The finding challenges conventional views of how genetic information is translated and points to a previously unrecognized mechanism that could influence cancer progression and response to targeted therapies.

The process by which cells turn genes into proteins has long been understood as precise and tightly controlled. But new research shows that cells can unexpectedly cut RNA into shorter fragments that still produce functional proteins, sometimes with harmful consequences.

Our immune systems never stop targeting cells they regard as a threat, so it’s really bad news when rogue immune cells mistakenly turn on us, as they do in autoimmune conditions. Existing treatments suppress these attacks, but don’t stop them. But a new approach that addresses the cause of these disorders by killing off the rogue cells is proving wildly successful.

“All the big pharma companies are jumping on the bandwagon now,” says Reuben Benjamin at King’s College London. There are dozens of clinical trials under way around the world, and the first treatments could be approved as early as next year, he says, as they’re proving to be vastly superior to those currently used.

The key to these new treatments are genetically engineered cells known as CAR T-cells. These are made from the T-cells that your immune system usually employs to kill off invasive bacteria or virus-infected cells. The T-cells are extracted from a person, programmed to attack a specific kind of cell and then returned to that individual.

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A revolutionary cancer treatment is now being applied to a wide range of autoimmune disorders. Columnist Michael Le Page finds it is proving to be even more effective than expected.

By Michael Le Page